Brain tumors are currently the leading cause of cancer-related mortality and morbidity in children. Glioblastoma multiforme (GBM) is a highly aggressive brain tumor and the first cancer to be comprehensively profiled by The Cancer Genome Atlas (TCGA) consortium. While GBM is less common in a pediatric setting than in adults, affected children show dismal outcomes similar to adult patients, and the vast majority will die within a few years of diagnosis despite aggressive therapeutic approaches. Tumors arise de novo (primary GBM) and are morphologically indistinguishable from their adult counterparts. A number of comprehensive studies have identified transcriptome-based subgroups and indicator mutations in adult GBM, and have thus enabled its molecular sub-classification. In contrast, while it has been demonstrated the presence of distinct molecular subsets of childhood GBM and described different genetic alterations compared to adult cases, the pediatric disease remains understudied. There is currently insufficient information to improve disease management, and since conventional treatments universally fail, there is a crucial need to identify relevant targets for the design of novel therapeutic agents.
It would be highly desirable to be provided with a biological marker for the prognostic and diagnostic of proliferation-associated disorders such as cancer. It would also be highly desirable to be provided with a potential candidate target for evaluating the usefulness of agents in the prevention, treatment and/or alleviation of symptoms associated with a proliferation-associated disorder. It would further be desirable to be provided with a novel therapy for the prevention, treatment and/or alleviation of symptoms associated with a proliferation-associated disorder.